IOM Urges CIRM to Develop Sustainability Plan

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California’s stem cell agency needs to develop a plan for sustaining itself when its $3 billion in state bond funding from Proposition 71 runs out in 2017, an agency-commissioned $700,000 study released yesterday urged.

The study, by a committee of the Institute of Medicine (IOM), also recommends that the California Institute for Regenerative Medicine (CIRM) increase the role of industry executives in the governing board and key aspects of the agency; tighten conflict-of-interest rules; and shift decisions on grant approvals and other operations from the governing board to day-to-day execs using advice from a working group.

CIRM commissioned the study in 2011, after the governing board or Independent Citizens Oversight Committee (ICOC) agreed with its then-chairman Robert Klein that IOM’s findings might encourage voters to approve another bond proposition for the agency.

In its 124-page report, “The California Institute for Regenerative Medicine: Science, Governance, and the Pursuit of Cures,” IOM recommended CIRM develop a “sustainability plan” detailing how it will fund its operations. Additional state funding is uncertain at best.

“We think CIRM ought to develop some different scenarios, assuming different things regarding just how the state of California will or will not continue its support, and at what level,” says the IOM committee’s chair Harold T. Shapiro.

Looking to Massachusetts

Shapiro, who is president emeritus at Princeton University and professor of economics and public affairs at the school's Woodrow Wilson School of Public and International Affairs, said one such scenario might be emulating the Boston-Cambridge region, which has enjoyed both $1 billion of state bond funds from the 2008 Massachusetts Life Sciences Act and at least as much in private dollars from investors and foundations.

The ICOC on May 24 adopted a CIRM strategic plan for 2012–2016. That plan calls for CIRM to push more of the science it funds into clinical trials, accelerate understanding of stem cell science, promote economic development for California, and position the state as a world leader in stem-cell science.

In the strategic plan, CIRM said it would “establish a platform to enable grantees, industry, other government agencies, disease foundations, venture capitalists, and others to continue to pursue CIRM’s mission upon the expiration of CIRM’s bond funding.”

The 2012 plan also commits CIRM to one-year and five-year clinical goals that the IOM says were overly ambitious. Within a year, for example, CIRM will ensure that its portfolio includes at least two programs with an approved IND filing with FDA to enter human clinical trials. Within five years, CIRM expectations include that “investigators will have established proof-of-principle in preclinical animal models for treatment of >10 diseases.”

“The translational goals enumerated in the 2012 strategic plan are unrealistic,” the IOM committee declared. “CIRM should also focus on fundamental biological mechanisms that ultimately determine the success or failure of a specific disease intervention and on the careful design of translational studies to make them maximally informative even in the absence of any demonstrable clinical benefit.”

To boost translation, CIRM on July 26 approved spending $151 million for eight new multidisciplinary “disease teams” charged with either filing an IND with FDA to begin clinical trials, or completing a Phase I/II clinical trial within four years. The agency has 22 disease teams with total funding of about $360 million.

Engaging industry

IOM did agree with CIRM’s strategic plan goal of increasing its share of funding for stem cell businesses from the current 6%. Only 15 for-profit entities have received CIRM grants and loans totaling around $80 million, though the agency has awarded just over half its $3 billion. California’s $114 billion, 2,240-company biopharma industry has long clamored for CIRM to fund more industry research.

Where CIRM’s plan envisioned separate industry and clinical boards, IOM recommended the agency create a single advisory board to discuss how to engage industry. IOM suggested CIRM study further its plan to attract industry through new “alpha” stem cell clinics that would deliver stem cell based therapies to patients, saying they could be integrated into existing academic medical centers.

IOM also recommended that CIRM recognize conflicts of interest arising from nonfinancial interests, such as an individual’s interest in a specific disease, and reassess policies for managing them. The report said the agency’s 29-member board should be restructured to give independent members a majority, and should limit its roles to strategic planning, finances, and oversight of CIRM president Alan Trounson, Ph.D.